Related and unrelated donor transplantation for β-thalassemia major: results of an international survey

Eapen, Mary ; Walters, Mark C. ; Smith, Angela R. ; Shenoy, Shalini ; Kasow, Kimberly ; Hanna, Rabi ; Goyal, Rakesh K. ; Auletta, Jeffery J. ; Agarwal, Rajni ; Dvorak, Christopher C. ; Boelens, Jaap Jan ; Keesler, Daniel A. ; Zhu, Yiping ; Li, Changgang ; Jiang, Hua ; Hebert, Kyle ; George, Biju ; Kim, Soyoung ; Mathews, Vikram ; Li, Chunfu (2019) Related and unrelated donor transplantation for β-thalassemia major: results of an international survey Blood Advances, 3 (17). pp. 2562-2570. ISSN 2473-9529

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Official URL: http://doi.org/10.1182/bloodadvances.2019000291

Related URL: http://dx.doi.org/10.1182/bloodadvances.2019000291

Abstract

We studied 1110 patients with β-thalassemia major aged ≤25 years who received transplants with grafts from HLA-matched related (n = 677; 61%), HLA-mismatched related (n = 78; 7%), HLA-matched unrelated (n = 252; 23%), and HLA-mismatched unrelated (n = 103; 9%) donors between 2000 and 2016. Ninety percent of transplants were performed in the last decade. Eight-five percent of patients received ≥20 transfusions and 88% were inadequately chelated. All patients received myeloablative-conditioning regimen. Overall and event-free survival were highest for patients aged ≤6 years and after HLA-matched related and HLA-matched unrelated donor transplantation. The 5-year probabilities of overall survival for patients aged ≤6 years, 7 to 15 years, and 16 to 25 years, adjusted for donor type and conditioning regimen were 90%, 84%, and 63%, respectively (P < .001). The corresponding probabilities for event-free survival were 86%, 80%, and 63% (P < .001). Overall and event-free survival did not differ between HLA-matched related and HLA-matched unrelated donor transplantation (89% vs 87% and 86% vs 82%, respectively). Corresponding probabilities after mismatched related and mismatched unrelated donor transplantation were 73% vs 83% and 70% vs 78%. In conclusion, if transplantation is considered as a treatment option it should be offered early (age ≤6 years). An HLA-matched unrelated donor is a suitable alternative if an HLA-matched relative is not available.

Item Type:Article
Source:Copyright of this article belongs to The American Society of Hematology.
ID Code:124122
Deposited On:04 Nov 2021 06:17
Last Modified:04 Nov 2021 06:17

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